Here’s a question more pharma executives are asking: Does patient engagement move the needle, or is it just good optics?

It’s a fair question.

In an era where pharmaceutical innovation must prove its value not only through clinical efficacy but also through demonstrated patient relevance, the question is no longer ‘whether’ to engage patients—it’s whether that engagement translates into an advantage.

New research from FleishmanHillard’s Global Health & Life Sciences group found it might. Released in recognition of Rare Disease Day, The Patient Engagement Premium: Defining the Strategic Value of Patient Input in Drug Development examines FDA submissions for rare disease therapies approved between 2018 and 2024 and finds directional associations between documented patient input and regulatory outcomes.

From Philosophy to Evidence

The shift from transactional patient engagement to embedded patient evidence isn’t new thinking though, but it is accelerating practice. And as regulatory scrutiny of traditional DTC channels intensifies and Health Technology Assessment bodies increasingly consult patient advocacy organizations, companies face a choice: embed patient evidence directly into development processes, or risk losing ground to those who do.

But let’s be honest, executive decision-makers demand more than anecdote. This research represents a crucial step toward establishing a measurable evidence base for patient engagement as a strategic investment, not just a values statement.

A Rigorous Approach to a Complex Question

The analysis examined 179 rare disease drug approvals that included Patient Experience Data (PED) tables, a requirement formalized following the 21st Century Cures Act. Each product was assigned a ‘Patient Engagement Score’ based on six distinct engagement activities, from patient advisory committee insights to patient-reported outcomes (PROs) and clinical outcome assessments (COAs).

Here’s what we found:

Patient input is increasingly embedded in regulatory submissions. Nearly nine in ten submissions in 2023-2024 explicitly cited at least one patient engagement activity, up markedly from earlier in the study period. PRO and COA data have become the most common form of patient input, signaling that companies may be integrating patient insights systematically and earlier in development.

Higher engagement scores trended with patient-centered labeling. Products with label claims tied to patient input averaged 1.4 documented engagement categories versus 1.0 for those without, a modest but directional association that could confer commercial advantage.

Company size isn’t a barrier. Mid-cap sponsors engaged in patient-centered activities nearly as frequently as large pharmaceutical companies. Translation? The potential benefits of patient engagement appear accessible across the competitive landscape.

What Happens to Companies That Don’t Move?

Let’s be clear: the evidence base is still developing, and these associations are directional rather than conclusive. But the implications are hard to ignore.

Patient engagement is evolving from ethical consideration to strategic necessity. Companies are prioritizing structured, quantifiable patient data, particularly PROs and COAs, for FDA submissions. Yet many underutilize other pathways, including patient organization partnerships and patient preference studies. That suggests that comprehensive investment in the full spectrum of patient evidence could be an untapped competitive edge.

For smaller companies not yet systematically integrating patient perspectives, the takeaway is encouraging, structured engagement may level the playing field. For larger companies that under-invest in patient input, the risk is equally clear, patient-centered rivals may be building advantages that compound over time.

Looking Ahead

As the evidence base expands and sponsors document patient engagement more comprehensively, clearer patterns will likely emerge. But the direction of travel is already obvious: regulators, payers, and patients themselves are reshaping how innovation is valued. Companies that embed patient engagement as foundational, not peripheral, will compound advantage across regulatory, payer and reputation landscapes. The infrastructure to do it exists. The question is in the execution.

Our approach combines regulatory expertise with data science and AI tools to help clients operationalize patient input across the product lifecycle, ensuring innovation is positioned as both evidence-driven and human-centered.

The pharmaceutical industry is at an inflection point. The companies that treat patient engagement as foundational—not peripheral—will define what comes next.

To access the full report or discuss how strategic patient engagement can create value for your organization, visit fleishmanhillard.com or contact Barry Sudbeck and Laura Musgrave, Patient Engagement Specialists with FleishmanHillard’s Global Health & Life Sciences group.